The Child-Centered Outcomes in Practice and Research (COPR) Center of Excellence: Strengthening the Clinical Validity Evidence Base for PROMIS Measures in Chronically Ill Children

 描述（由申请人提供）：患者报告结果（PRO）工具通过从患者的角度检查他们的感受、他们认为自己可以做什么、他们认为自己可以参与哪些类型的活动以及他们对自己的健康和生活的满意度来补充传统的临床结果。为了推进 PRO 评估的科学性，NIH 于 2004 年推出了患者报告结果测量信息系统 (PROMIS(r))。尽管 PROMIS 的一个关键目标是为整个生命周期的研究和临床实践提供 PRO，但人们对 PROMIS 对患有慢性疾病的儿童的有效性或 PROMIS 检测到儿童健康方面有临床意义的变化的程度知之甚少。为了满足这些科学需求，我们建议创建以儿童为中心的实践和研究成果 (COPR) 卓越中心，该中心将致力于严格检查和记录 PROMIS 对慢性病儿童的临床有效性。 COPR 包括三个研究项目，重点关注儿科克罗恩病、慢性肾病和癌症。这些项目通过儿童体验幸福感（一个包含痛苦和幸福的概念）、临床验证方法和基础设施的共同衡量标准联系在一起。每个项目将评估 PROMIS 儿科疼痛干扰、疲劳、心理压力体验和积极情感测量的内容效度、评分的临床意义、对临床变化的反应性以及纵向结构效度。 COPR 方法将根据 PROMIS 标准提高测量的心理测量和临床成熟度，并提供 FDA 所需的证据，使 PRO 仪器能够进行临床试验。 COPR 团队整合了： 一个 PROMIS 研究小组，该小组开发了 20 项现有 PROMIS 儿科措施中的 10 项，并在过去 6 年里帮助领导了 PROMIS 计划；由 PCORI 资助的临床数据研究网络 (PEDSnet)，在电子健康记录和 PRO 数据管理、质量评估和报告方面拥有深厚的专业知识；一个由儿科胃肠病学家组成的全国网络，开始开展 PCORI 资助的针对克罗恩病儿童的临床试验；一个全国儿科肾病专家网络，正在对患有慢性肾病的儿童进行一项由 NIH 资助的队列研究；以及位于圣裘德儿童研究医院的全国最大的儿科肿瘤临床中心之一。 COPR 基础设施将： 促进三个研究项目之间的协调；标准化临床验证方法和信息资源；综合三个项目的结果；开发基于标准的仪器档案，以加强 PROMIS 的临床采用，并促进 FDA 对 PROMIS 措施作为临床试验终点的资格认证；支持与其他 PEPR 研究项目的合作。 COPR 经过优化以成功完成其三个研究项目，并且其基础设施可以轻松扩展以支持 多个其他 PEPR 研究计划的信息学、统计和管理需求。

 DESCRIPTION (provided by applicant): Patient-reported outcome (PROs) instruments complement conventional clinical outcomes by examining from the perspective of patients how they feel, what they perceive they can do, which types of activities they believe they can participate in, and how satisfied they are with their health and lives. To advance the science of PRO assessment, the NIH launched in 2004 the Patient Reported Outcome Measurement Information System (PROMIS(r)). Although a key objective of PROMIS is to provide PROs for research and clinical practice across the life course, there is remarkably little known about the validity of PROMIS for children with chronic diseases or the degree to which PROMIS detects clinically meaningful changes in children's health. To address these scientific needs, we propose to create the Child-Centered Outcomes in Practice and Research (COPR) Center of Excellence, which will be devoted to rigorously examining and documenting PROMIS' clinical validity for children with chronic conditions. COPR comprises three research projects focusing on pediatric Crohn's disease, chronic kidney disease, and cancer. The projects are linked by common measures of children's Experienced Well-Being (a concept that subsumes both suffering and happiness), clinical validation methods, and infrastructure. Each project will assess the content validity, clinical meaningfulness of scores, responsiveness to clinical change, and longitudinal construct validity of the PROMIS Pediatric pain interference, fatigue, psychological stress experiences, and positive affect measures. The COPR approach will increase the measures' psychometric and clinical maturation according to PROMIS standards and provide evidence required by the FDA to qualify PRO instruments for clinical trials. The COPR team integrates: a PROMIS research group that developed 10 of the 20 extant PROMIS pediatric measures and helped lead the PROMIS initiative over the past 6 years; a PCORI-funded clinical data research network (PEDSnet) with deep expertise in electronic health record and PRO data management, quality assessment and reporting; a national network of pediatric gastroenterologists embarking on a PCORI-funded clinical trial for children with Crohn's disease; a national network of pediatric nephrologists who are conducting an NIH-funded cohort study on children with chronic kidney disease; and, one of the nation's largest pediatric oncology clinical centers located at St. Jude Children's Research Hospital. The COPR infrastructure will: facilitate coordination across its three research projects; standardize clinicl validation methods and informatics resources; synthesize results across the three projects; develop standards-based instrument dossiers to enhance clinical adoption of PROMIS and facilitate FDA qualification of PROMIS measures as clinical trial end-points; and, support collaboration with other PEPR research programs. COPR is optimized to successfully accomplish its three research projects, and its infrastructure can be readily scaled to support the informatics, statistical, and administrative needs of multiple other PEPR research initiatives.